Alerta

COMPOUND FOR RECOGNIZING α-SYNUCLEIN AGGREGATE, AND USE THEREOF

Resumen de: WO2025162452A1

A compound specifically binding to an α-synuclein aggregate, and a preparation method therefor and the use thereof. Specifically, the compound binding to the α-synuclein aggregate comprises compounds as shown in formula A or sub-general formulas thereof, or stereoisomers, pharmaceutically acceptable salts, solvates or stable isotope variants thereof. The compound is a small molecule tracer, which can specifically recognize the α-synuclein aggregate, and can be used for the preparation of a drug for the treatment or diagnosis of neurodegenerative diseases (such as Parkinson's disease, dementia with Lewy bodies, multiple system atrophy, Alzheimer's disease, amyotrophic lateral sclerosis, progressive supranuclear palsy and progressive muscular atrophy) related to the α-synuclein aggregate and other misfolded proteins.

EDARAVONE SUSPENSION FOR ORAL ADMINISTRATION

Resumen de: AU2025205635A1

This invention provides with an edaravone suspension for oral administration having excellent bioavailability. It is expected that burden on ALS patients and care workers can be reduced thereby. This invention provides with an edaravone suspension for oral administration having excellent bioavailability. It is expected that burden on ALS patients and care workers can be reduced thereby. ul h i s i n v e n t i o n p r o v i d e s w i t h a n e d a r a v o n e s u s p e n s i o n f o r o r a l a d m i n i s t r a t i o n h a v i n g e x c e l l e n t u l b i o a v a i l a b i l i t y t i s e x p e c t e d t h a t b u r d e n o n p a t i e n t s a n d c a r e w o r k e r s c a n b e r e d u c e d t h e r e b y

FTO INHIBITORS OF BICYCLIC STRUCTURES

Resumen de: WO2025162358A1

Disclosed herein is a compound of Formulas (I) and (II) as an FTO inhibitor with novel structures. Also disclosed herein is a pharmaceutical composition comprising the same, and a method of inhibiting weight gain, promoting weight loss, reducing serum LDL, cholesterol, LDL-c, or triglycerides, or treating obesity or an obesity-related disease (esp. obesity-related diabetes, hyperglycemia, diabetic nephropathy, hyperlipemia, coronary heart disease, atherosclerosis, hypertension, cardiovascular or cerebrovascular disease) or Alzheimer's disease by inhibiting FTO by using the compound disclosed herein.

C-17 CARBONYL-SUBSTITUTED OLEANANE TRITERPENE DERIVATIVE, PREPARATION METHOD THEREFOR, AND USE THEREOF

Resumen de: WO2025162103A1

The present invention relates to a C-17 carbonyl-substituted oleanane triterpene derivative, a preparation method therefor, and a use thereof. Provided are a C-17 carbonyl-substituted oleanane triterpene derivative, a use of the compound in the preparation of an NRF2 activator, and preparation of a medicament for treating/preventing diseases. The diseases comprise cerebral small vascular disease, mitochondrial encephalomyopathy, autism spectrum disorder, Rett syndrome, Friedreich ataxia, stroke, hemorrhagic cerebral apoplexy, ischemic cerebral apoplexy, multiple sclerosis, amyotrophic lateral sclerosis, schizophrenia, schizophrenic cognitive impairment, Parkinson's disease, cognitive impairment in Parkinson's disease, Alzheimer's disease, vascular dementia, epilepsy, Huntington's disease, heart failure, myocardial infarction, renal failure, kidney ischemia, etc., or other disease states and conditions which are obvious to a person skilled in the art. Also provided are a prodrug thereof, or a pharmaceutically acceptable salt thereof, or a hydrate or solvate thereof, and a pharmaceutical composition containing same.

C17-SITE NITROGEN-SUBSTITUTED AND METHYLENE-SUBSTITUTED OLEANANE TRITERPENE DERIVATIVE, PREPARATION METHOD THEREFOR AND USE THEREOF

Resumen de: WO2025162101A1

A C17-site nitrogen-substituted and methylene-substituted oleanane triterpene derivative, a preparation method therefor, and a use thereof. Provided are a C17-site nitrogen-substituted and methylene-substituted oleanane triterpene derivative, a use of the compound in the preparation of an NRF2-Leap1 decoupling agent, and a use of the compound in the preparation of a medicament for preventing and/or treating diseases in a patient, the diseases comprising cerebral small vessel disease, mitochondrial encephalomyopathy, autism spectrum disorder, Rett syndrome, Friedreich ataxia, stroke, hemorrhagic cerebral apoplexy, ischemic cerebral apoplexy, multiple sclerosis, amyotrophic lateral sclerosis, schizophrenia, cognitive impairment in schizophrenia, Parkinson's disease, cognitive impairment in Parkinson's disease, Alzheimer's disease, vascular dementia, epilepsy, Huntington's disease, heart failure, myocardial infarction, renal failure, kidney ischemia, etc.

ACETYL-LEUCINE FOR TREATING PARKINSON ́S DISEASE

Resumen de: WO2025163129A1

The present disclosure provides for treating Parkinson ́s disease (PD) comprising administering acetyl-leucine or a pharmaceutically acceptable salt thereof to a subject in need thereof.

Variant RNAi

Resumen de: AU2025205501A1

18808542_1 (GHMatters) P43228AU01 Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi for treating Huntington’s disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington’s disease. Provided herein are RNAi molecules including a first strand containing a guide sequence and a second strand comprising a non-guide sequence where the non-guide sequence contains a bulge opposite the seed region of the guide sequences; e.g., opposite the cleavage sequence. In some aspects, the invention provides RNAi for treating Huntington's disease. Further provided herein are expression cassettes, vectors (e.g., rAAV, recombinant adenoviral, recombinant lentiviral, and recombinant HSV vectors), cells, viral particles, and pharmaceutical compositions containing the RNAi. Yet further provided herein are methods and kits related to the use of the RNAi, for example, to treat Huntington's disease. 18808542_1 (GHMatters) P43228AU01 ul u l r o v i d e d

ARYL HETEROCYCLIC KV1.3 INHIBITOR, AND PREPARATION METHOD THEREFOR AND USE THEREOF

Resumen de: WO2025161144A1

The present invention provides a novel Kv1.3 channel (or Kv1.3) inhibitor, which can be used for preventing and/or treating Kv1.3 channel (or Kv1.3)-related diseases, including immune and inflammatory diseases, such as multiple sclerosis, inflammatory bowel disease, ulcerative colitis, Crohn's disease, rheumatoid arthritis, type I diabetes, psoriasis and asthma, spondylitis and periodontitis; and obesity, type 2 diabetes, renal fibrosis, Alzheimer's disease, and ischemic stroke.

FOOD- OR FEEDSTUFF COMPOSITION WITH EFFECT ON NEURODEGENERATIVE DISEASES

Resumen de: WO2025162734A1

The present invention relates to a food- or feedstuff composition comprising a B. subtilis strain and a Ginkgo biloba extract, such composition for use as a medicament as well as such composition for use in treating or preventing neurodegenerative diseases, in particular Alzheimer's disease as well as the use of such composition as a food supplement.

USE OF MESENCHYMAL-STEM-CELL-DERIVED INTRACELLULAR NANOVESICLE IN NEUROPROTECTION

Resumen de: WO2025162163A1

Disclosed in the present invention is the use of a mesenchymal-stem-cell-derived intracellular nanovesicle in neuroprotection. Compared to a small extracellular vesicle with exosomes as the main component, the small intracellular nanovesicle of the present invention has a smaller particle size, a narrower particle size distribution range, and greater stability at different temperatures, and has good tissue compatibility. The small intracellular nanovesicle of the present invention can better ameliorate nerve injury or neurodegenerative diseases such as optic nerve injury, ischemic stroke and Alzheimer's disease, and has very good application and research value in the field of pharmaceuticals.

NOVEL ALPK1 INHIBITORS

Resumen de: AU2024209890A1

The disclosure is directed to novel ALPK1 inhibitors having the Formula (I), or a pharmaceutical acceptable salt, a stereoisomer, a tautomer, a stable isotopic variant, a prodrug, or a crystal form thereof. The disclosure is also directed to pharmaceutical composition comprising the novel ALPK1 inhibitors, and use thereof in treating inflammation related diseases, such as ROSAH syndrome, inflammatory bowel disease (IBD), NASH, gout, diabetes, chronic kidney disease, pancreatitis, Kawasaki disease, inflammatory skin diseases and neurodegenerative diseases including the Alzheimer's disease.

TREATMENT OF PARKINSON'S DISEASE AND PARKINSON'S DISEASE PSYCHOSIS

Resumen de: US2025248985A1

The invention relates generally to the treatment of Parkinson's Disease (PD), including Parkinson's Disease psychosis (PDP) with iloperidone.

Methods for Providing Rapid Relief of Motor Fluctuations in a Parkinson's Disease Patient

Resumen de: US2025248960A1

The present invention provides methods of providing rapid relief of motor fluctuations in a Parkinson's disease patient. The methods of the invention comprise pulmonary administration of levodopa by inhalation at therapeutically effective concentrations such that the patient's plasma levodopa concentration increases by at least about 200 ng/ml within 10 minutes or less post inhalation as compared to the concentration of levodopa in the patient's plasma prior to inhalation of the levodopa and wherein the patient's plasma concentration remains increased by at least about 200 ng/ml for a time period of at least 15 minutes after inhalation. The methods of the invention are particularly useful for treatment of motor fluctuations which arise as a result of poorly controlled levodopa plasma levels in a patient.

NOVEL FUSION PROTEIN FOR ELIMINATING NEURODEGENERATIVE DISEASE-CAUSING FACTOR AND USE THEREOF

Resumen de: US2025250324A1

A fusion protein of the present disclosure may induce the degradation of neurodegenerative disease-causing factors by binding to Tau and amyloid-beta proteins as the neurodegenerative disease-causing factors and activating autophagy. In addition, the fusion protein of the present disclosure penetrates a blood-brain barrier and is introduced into cells to be effectively delivered into nerve cells without a separate carrier, and has high stability and thus is expected to be used as a platform for the treatment of neurodegenerative diseases such as dementia and Alzheimer's disease.

LEVODOPA FATTY ACID DERIVATIVES, FORMULATIONS THEREOF, AND THEIR USES FOR THE TREATMENT OF PARKINSON'S DISEASE

Resumen de: EP4595956A1

A levodopa derivative including a compound or pharmaceutically acceptable salt, hydrate, and/or solvate thereof, wherein the compound includes substituents which, in aggregate, contain at least 6 carbon atoms which are only bonded to either other carbon atoms or to hydrogen atoms. The levodopa derivative may be formulated as a composition including one or more pharmaceutically acceptable carriers or excipients. The levodopa derivative may be part of a pharmaceutical composition including micro or nano particles in which the levodopa derivative is encapsulated in the pharmaceutically acceptable polymer. The levodopa derivative can be used to treat Parkinson's disease by administering to a mammal an amount sufficient to treat Parkinson's disease.

METHODS FOR TREATING EARLY ALZHEIMER'S DISEASE

Resumen de: CN120051284A

Provided herein are methods of treating early Alzheimer's disease using hydroxypropyl beta-cyclodextrin compositions.

COMPOSITIONS AND METHODS FOR THE TREATMENT OF NEUROLOGICAL DISORDERS RELATED TO GLUCOSYLCERAMIDASE BETA 1 DEFICIENCY

Resumen de: MX2025008267A

The disclosure relates to compositions and methods for altering, <i>e.g.</i>, enhancing, the expression of GCase proteins, whether <i>in vitro</i> and/or <i>in vivo</i>. Such compositions include delivery of an adeno-associated viral (AAV) particle. The compositions and methods of the present disclosure are useful in the treatment of subjects diagnosed with, or suspected of having Parkinson's Disease (PD), Gaucher Disease (GD), Dementia with Lewy Bodies (DLB), or related condition resulting from a deficiency in the quantity and/or function of GBA1 gene product or associated with decreased expression or protein levels of GCase protein.

PRIDOPIDINE FOR TREATING JUVENILE HUNTINGTON'S DISEASE

Resumen de: MX2025007590A

Provided herein a method of treating Juvenile Huntington disease in a subject in need thereof comprising orally administering a pharmaceutical composition comprising pridopidine and/or its analog or a pharmaceutically acceptable salt thereof.

ANTI-AMYLOID β PROTOFIBRIL/OLIGOMER ANTIBODIES AND USES THEREOF

Resumen de: MX2025008034A

The present disclosure provides anti-amyloid β (Aβ) antibodies and antibody fragments that preferentially bind soluble amyloid Aβ protofibril/oligomer and trigger ADPC in microglial cells, anti-amyloid β (Aβ) antibodies and antibody fragments that reduce soluble amyloid Aβ protofibril/oligomer levels and insoluble amyloid Aβ plaque in brain tissue, and the use of anti-Aβ protofibril/oligomer antibodies and antibody fragments in therapy, prophylaxis, diagnosis, screening, and monitoring of conditions associated with Aβ protein aggregation, in particular Alzheimer's disease (AD).

4-AMINOPYRROLO2,1-F1,2,4TRIAZINES AND PREPARATION AND USES THEREOF

Resumen de: MX2025005198A

4-Aminopyrrolo2,I-f1,2,4triazine compounds of formula I for treating various diseases and pathologies are disclosed. More particularly, the present disclosure concerns the use of 4- aminopyrrolo2,1-fl,2,4triazine compounds or analogs thereof, in the treatment of disorders characterized by overexpression of DYRK1A (e.g., cancer, Down syndrome, Alzheimer's disease, diabetes, and osteoarthritis).

NOVEL APOE ANTISENSE OLIGONUCLEOTIDE AND USE THEREOF

Resumen de: WO2025159427A1

The present invention relates to: an apolipoprotein E (APOE) antisense oligonucleotide; and a pharmaceutical composition for treating Alzheimer's disease comprising same. The antisense oligonucleotide of the present invention can reduce the expression of the APOE4 variant gene that contributes to increased risk and exacerbation of Alzheimer's disease, and can be used as an RNA therapeutic agent for diseases caused by abnormal levels of APOE proteins or APOE variant genome expression.

NLRP3 INFLAMMASOME INHIBITORS AND COMPOSITIONS AND USES THEREOF

Resumen de: US2025243172A1

NLRP3 selective inhibitors (NSIs) as anti-inflammatory agents are provided, as are methods of using NSIs to inhibit inflammation and prevent or treat diseases and conditions associated with inflammation, such as Alzheimer's disease, Parkinson's disease, multiple sclerosis, traumatic brain injury, acute myocardial infarction, heart failure, arthritis, diabetes, gout, COVID-19, and autoinflammatory diseases.

ANTI-FSH ANTIBODIES FOR NEURODEGENERATIVE DISEASES

Resumen de: US2025243269A1

The present disclosure provides compositions and methods for treating neurodegenerative diseases, in particular, Alzheimer's Disease, by using anti-FSH antibodies in a subject in need thereof. In some embodiments, the subject has a condition in which FSH levels are elevated. The methods include administering to said subject a therapeutically effective amount of an anti-FSH antibody or an antigen-binding portion thereof.

CHIMERIC ANTIGEN RECEPTOR MACROPHAGE COMPOSITIONS AND USES THEREOF

Resumen de: US2025241950A1

Provided are chimeric antigen receptor (CAR) that bind to beta amyloid, macrophages (CAR-Ms) that express the CAR, and compositions comprising the same. Also provided are methods for reducing one or more symptoms associated with Alzheimer's disease using the CAR-Ms.

METHODS OF TREATING AMYOTROPHIC LATERAL SCLEROSIS BY ORAL ADMINISTRATION OF FASUDIL

Nº publicación: WO2025160116A1 31/07/2025

Solicitante:

WOOLSEY PHARMACEUTICALS INC [US]
WOOLSEY PHARMACEUTICALS, INC

Resumen de: WO2025160116A1

A method includes treatment of a sporadic ALS patient with oral fasudil at a dose exceeding 240 mg/day. This results in an anticipated 25-50% reduction in the average decline over at least three months as measured using the revised ALS Functional Rating Scale.